Research and Application of Antisense Oligonucleotide in the Treatment of Nervous System Diseases
The etiology and pathological mechanisms underlying nervous system diseases are intricate,posing challenges in pinpoint-ing drug targets for the development of novel therapeutics.Advances in genome and transcriptome sequencing,along with other techno-logical innovations,have facilitated significant strides in uncovering targets and molecular pathways implicated in genetic,non-hereditary,or sporadic nervous system disorders.The emergence of small nucleic acid drugs,based on transcriptome-target discovery,represents a promising avenue for addressing these diseases.Among these,antisense oligonucleotide(ASO)constitute a subclass of small nucleic acid drugs.ASO,which target mRNA,offer several advantages over traditional small molecule or antibody-based therapies that target proteins,including a broad spectrum of candidate targets,expedited target screening,and a heightened success rate in drug development.At present,the treatment of ASO drugs in the field of the realm of nervous system disorders is widely concerned by numer-ous pharmaceutical research and development enterprises,and ASO drugs has a good prospect of clinical application.This review summa-rizes the research and clinical applications of ASO in treating diverse nervous system pathologies,elucidating their mechanism of action,developmental advantages,structural modification methodologies,and the latest advancements in clinical application for the treatment of nervous system diseases,to provide reference for the research and development of new drugs for neurological disorder,especially for rare and refractory diseases.
Antisense oligonucleotideSmall nucleic acid drugsNervous system diseasesResearch progress
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